보고서 요약(국문)

개요
전 세계 척수근육위축증(SMA) 치료 시장은 2022년 XX백만 달러에 달했으며, 2023년부터 2030년까지 연평균 XX%의 성장률을 기록하며 2030년에는 XX백만 달러에 이를 것으로 예상됩니다.
척수근육위축증(SMA)은 근육이 약해지는 유전성 신경근육 질환입니다. SMA 환자는 척수에서 근육 운동을 조절하는 운동 신경 세포라는 특정 유형의 신경 세포를 잃게 됩니다. 이 운동 신경 세포가 없으면 근육은 움직임을 유발하는 신경 신호를 받지 못합니다. '위축'이라는 의학 용어는 '작아짐'을 의미합니다. SMA에서는 특정 근육이 사용 부족으로 인해 작아지고 약해집니다.
척수근육위축증은 완치할 수 없습니다. 치료는 SMA 유형과 증상에 따라 다릅니다. 많은 SMA 환자들이 물리 치료, 작업 치료, 그리고 정형외과용 보조기, 목발, 보행기, 휠체어와 같은 보조 기구의 도움을 받습니다. SMA는 주로 영유아와 어린이에게 발생하지만 성인에게도 나타날 수 있습니다. 척수근육위축증(SMA)의 유형별로 증상과 예후는 다양합니다. 유전자 대체 요법과 질병 진행 억제 요법은 희망을 제시합니다.
클리블랜드 클리닉(Cleveland Clinic)에 따르면, 미국에는 약 1만 명에서 2만 5천 명의 어린이와 성인이 척수근육위축증을 앓고 있습니다. 이는 6천 명에서 1만 명의 어린이 중 한 명꼴로 발생하는 희귀 질환입니다.
시장 동향: 성장 동력
신규 치료제 도입 증가
신규 치료제 도입 증가는 예측 기간 동안 시장 성장을 견인할 것으로 예상됩니다. 척수근육위축증 치료법은 전령 RNA(pre-mRNA) 스플라이싱을 조절하고 이 질환에서 발현 수준이 낮은 생존운동신경세포(SMN) 단백질의 발현을 증가시켜 질병의 세포적 기전을 차단하도록 설계되었습니다. 이러한 원리를 기반으로 새로운 치료제가 개발되고 있으며, 이에 따라 신규 치료제에 대한 수요가 증가하고 있고, 이러한 치료제의 도입이 늘어나 환자 예후가 개선되고 있습니다.

예를 들어, 2023년 12월 시카고 앤 앤 로버트 H. 루리 어린이 병원의 스탠리 맨 어린이 연구소에 있는 마용차오 박사 연구팀은 척수근육위축증(SMA)에서 운동 신경 세포 퇴화를 유발하는 새로운 기전을 발견했습니다. 이 발견은 SMA에 대한 유전자 치료 및 기타 기존 치료법의 중요한 한계를 극복하는 새로운 치료 표적을 제시합니다.
또한, 2023년 12월 홍콩 병원관리국(HA)은 척수근육위축증(SMA)을 앓는 영아를 위한 유전자 치료 주사를 도입했는데, 이는 기존 치료법보다 효과적입니다. 홍콩에서는 10개월 된 영아가 오나셈노젠 아베파르보벡(onasemnogene abeparvovec)이라는 이 치료를 처음으로 받았다고 발표했습니다.
졸겐스마(Zolgensma)와 같은 새로운 치료제는 임상 시험에서 놀라운 효능을 보여주었으며, SMA 환자의 운동 기능과 삶의 질을 크게 향상시키는 경우가 많습니다. 이는 의료 전문가와 환자 모두의 신뢰와 수용도를 높이는 데 기여했습니다. 또한, 질환 치료 효과를 높이기 위한 더욱 발전된 신규 치료법 개발을 위한 임상 시험이 진행 중입니다.
예를 들어, 2023년 9월 14일, 다양한 희귀 질환 및 일반 질환 치료를 위한 혁신적인 치료법의 발굴, 개발 및 상용화에 주력하는 글로벌 임상 단계 바이오제약 회사인 바이오헤이븐(Biohaven Ltd.)은 척수근육위축증(SMA) 환자를 대상으로 한 탈데프그로베프 알파(taldefgrobep alfa)의 3상 임상 시험인 레질리언트(RESILIENT) 연구의 환자 등록을 완료했다고 발표했습니다.
더 나아가, 척수근육위축증의 유병률 증가, 신규 치료법에 대한 FDA 승인 증가, 더욱 발전된 치료법 개발을 위한 임상 시험 증가, 질환에 대한 인식 제고 및 신규 치료법 개발의 진전은 예측 기간 동안 시장 성장을 견인할 것으로 예상되는 요인입니다.

제한 요인
다양한 치료법과 관련된 합병증 및 부작용, 높은 치료 비용, 충족되지 않은 의료 수요 및 치료 반응에 대한 불확실성은 시장 성장을 저해할 것으로 예상되는 요인입니다.

시장 세분화 분석
전 세계 척수근육위축증 치료 시장은 질병 유형, 치료 유형, 투여 경로, 최종 사용자 및 지역별로 세분화됩니다.
질병 조절 치료제 부문은 척수근육위축증 치료 시장 점유율의 약 45.6%를 차지했습니다.
질병 조절 치료제 부문은 예측 기간 동안 가장 큰 시장 점유율을 유지할 것으로 예상됩니다. 이러한 질병 조절 치료제는 SMN 단백질 생성을 촉진합니다. 누시너센(스핀라자)은 소아와 성인 모두에게 FDA 승인을 받았습니다. 의료진은 이 약물을 척수관 주변 공간에 주사합니다. 리스다플람(에브리스디)은 성인과 생후 2개월 이상 소아에게 사용됩니다. 리스다플람은 매일 경구 복용합니다.

예를 들어, 2020년 8월 7일, PTC Therapeutics, Inc.는 성인 및 2개월 이상 소아의 척수근육위축증(SMA) 치료를 위한 최초의 가정용 경구 투여 치료제인 에브리스디(리스디플람)에 대해 미국 식품의약국(FDA)의 승인을 받았습니다. 에브리스디는 2개월 이상 환자를 대상으로 한 두 건의 임상 시험에서 1형, 2형, 3형 SMA를 포함한 모든 질병 중증도 단계에서 운동 기능 및 발달 이정표 달성에 임상적으로 의미 있는 개선을 보였습니다.
또한, 2021년 7월, 로슈는 인도에서 SMA 환자 치료제로 승인된 에브리스디(리스디플람)를 출시했습니다. 미국 승인 후 11개월 만에 인도에서 시판되기 시작했습니다. 출시 이후 50개국 이상에서 4,000명 이상의 SMA 환자들이 에브리스디의 혜택을 받았습니다. 에브리스디는 성인 및 2개월 이상 소아의 SMA 치료제로 승인되었습니다. 또한, 리스디플람(에브리스디)은 폭넓은 보급, 수용성, 그리고 간편한 사용법 덕분에 가장 큰 시장 점유율을 차지하고 있습니다. 리스디플람(에브리스디)은 SMN2 유전자의 단백질 생성 저해를 차단하여 필요한 단백질이 신경 세포에 도달하도록 돕습니다. 복용량은 환자의 체중에 따라 결정됩니다. 임상 시험 결과, 12개월 후 복용자의 41%에서 근육 기능이 개선된 것으로 나타났습니다. 뿐만 아니라, 리스디플람의 광범위한 사용은 이러한 약물에 대한 수요 증가로 이어지고 있습니다.
예를 들어, 2021년 8월 12일, 주가이 제약은 척수근육위축증(SMA) 치료제인 에브리스디 건조 시럽 60mg을 출시했습니다. 이번 승인은 증상이 있는 1형 척수성 근육 위축증(SMA) 영아를 대상으로 한 FIREFISH 연구와 2형 또는 3형 SMA 소아 및 청소년을 대상으로 한 SUNFISH 연구 결과를 바탕으로 이루어졌습니다.
지역 분석
북미 지역은 시장 점유율의 약 41.4%를 차지했습니다.
북미 지역은 주요 업체들의 강력한 입지와 첨단 치료법 개발을 위한 연구 활동 증가로 인해 예측 기간 동안 가장 큰 시장 점유율을 유지할 것으로 예상됩니다. 특히 미국은 제약 회사와 같은 주요 업체들의 강력한 존재감으로 잘 알려져 있습니다. 이 지역의 주요 업체들은 SMA 치료를 위한 더욱 발전된 약물을 개발하기 위해 다른 회사들과 협력 및 합병을 진행하고 있습니다.

예를 들어, 신경유전학 의약품 개발에 전념하는 생명공학 기업인 보이저 테라퓨틱스(Voyager Therapeutics, Inc.)는 2024년 1월 2일, 헌팅턴병(HD)과 척수근육위축증(SMA)에 대한 잠재적 유전자 치료법 개발을 위해 노바티스 파마(Novartis Pharma AG)와 전략적 협력 및 캡시드 라이선스 계약을 체결했다고 발표했습니다. 보이저는 노바티스에 자사의 TRACER 캡시드 및 해당 질환 관련 기타 지적 재산권에 대한 독점적 접근 라이선스를 제공할 예정입니다.
또한, 척수근육위축증의 보다 효과적인 치료를 위한 연구 활동이 증가하고 있는 것도 이 지역 시장 성장을 견인하는 요인입니다. 연구 활동이 활발해짐에 따라, 기존 치료법과 관련된 합병증을 줄여 더욱 정밀한 치료제 개발이 이루어지고 있으며, 이는 환자 치료 결과 개선으로 이어지고 있습니다.

예를 들어, 2022년 3월 14일, 바이오젠(Biogen Inc.)은 스핀라자(누시너센)와 척수근육위축증(SMA) 연구 프로그램(ASCEND, RESPOND, NURTURE 연구 포함)에서 얻은 새로운 데이터와 업데이트를 발표했습니다. 이 프로그램은 SMA 환자의 임상 결과 개선을 목표로 합니다.
경쟁 환경
척수근육위축증 치료 시장의 주요 글로벌 업체로는 노바티스(Novartis AG), F. 호프만-라로슈(F. Hoffmann-La Roche Ltd), 제넨텍 USA(Genentech USA, Inc.), 바이오젠(Biogen Inc.), PTC 테라퓨틱스(PTC Therapeutics, Inc.), 아이오니스 파마슈티컬스(Ionis Pharmaceuticals, Inc.), 바이오헤이븐(Biohaven Ltd), 보이저 테라퓨틱스(Voyager Therapeutics, Inc.), 카탈렌트(Catalent, Inc.), 화이자(Pfizer Inc.) 등이 있습니다.
코로나19 영향 분석
코로나19 팬데믹은 전 세계 척수근육위축증 치료 시장에 상당한 영향을 미쳤습니다. 2019년 코로나바이러스 감염증(COVID-19) 팬데믹은 의료 환경의 재편을 초래했으며, 이는 척수근육위축증(SMA) 환자에 대한 임상 치료 제공에 영향을 미쳤습니다. SMA 환자는 COVID-19의 중증 증상을 나타낼 위험이 더 높을 수 있다는 우려가 있습니다. 현재 승인된 SMA 치료법은 생존율과 운동 기능을 향상시키지만, 이러한 치료법을 제공하려면 의료 시스템에 대한 노출이 증가하고 전문 의료진이 필요합니다.
시장 세분화
질병 유형별
• 0형 SMA (태아기 SMA)
• 1형 SMA (영아기 SMA, 베르드니히-호프만병)
• 2형 SMA (중간기 SMA, 두보위츠병)
• 3형 SMA (소아기 SMA, 쿠겔베르크-웰란더병)
• 4형 SMA (성인기 후기 발병 SMA)
치료 유형별
• 질병 진행 억제 치료
• 유전자 대체 치료
• 기타
투여 경로별
• 정맥 주사
• 척수강내 주사
• 경구 투여
• 기타
최종 사용자별
• 병원
• 전문 클리닉
• 학술 및 연구 기관
• 유전자 치료 센터
• 기타
지역별
• 북미
o 미국
o 캐나다
o 멕시코
• 유럽
o 독일
o 영국
o 프랑스
o 스페인
o 이탈리아
o 기타 유럽
• 남미
o 브라질
o 아르헨티나
o 남미 기타 지역
• 아시아 태평양
o 중국
o 인도
o 일본
o 호주
o 아시아 태평양 기타 지역
• 중동 및 아프리카
보고서 구매 이유

• 질병 유형, 치료 유형, 투여 경로, 최종 사용자 및 지역별 글로벌 척수근육위축증 치료 시장 세분화를 시각화하고 주요 상업 자산 및 플레이어를 파악하기 위해

• 트렌드 분석 및 공동 개발을 통해 사업 기회를 식별하기 위해
• 모든 세그먼트를 포함한 척수근육위축증 치료 시장 수준의 다양한 데이터가 담긴 Excel 데이터 시트 제공

• 심층적인 질적 인터뷰와 연구를 바탕으로 한 종합적인 분석이 담긴 PDF 보고서 제공

• 주요 플레이어의 핵심 제품을 모두 포함하는 제품 맵핑 Excel 파일 제공

글로벌 척수근육위축증 치료 시장 보고서는 약 69개의 표, 72개의 그림, 187페이지로 구성됩니다.

2023년 목표 고객층
• 제조업체/구매자
• 산업 투자자/투자 은행가
• 시장 조사 전문가
• 신흥 기업

보고서 요약(영어 원문)

Overview
Global spinal muscular atrophy treatment market reached US$ XX million in 2022 and is expected to reach US$ XX million by 2030, growing at a CAGR of XX% during the forecast period 2023-2030.
Spinal muscular atrophy (SMA) is a genetic (inherited) neuromuscular disease that causes muscles to become weak. People with SMA lose a specific type of nerve cell in the spinal cord called motor neurons that control muscle movement. Without these motor neurons, muscles don’t receive nerve signals that make muscles move. The word atrophy is a medical term that means smaller. With SMA, certain muscles become smaller and weaker due to lack of use.
There is no cure for spinal muscular atrophy. Treatments depend upon the type of SMA and symptoms. Many people with SMA benefit from physical and occupational therapy and assistive devices, such as orthopedic braces, crutches, walkers and wheelchairs. It mostly affects infants and children but can also develop in adults. Symptoms and prognosis vary depending on SMA type. Gene replacement and disease-modifying therapies offer hope.
According to the Cleveland Clinic Organization, approximately 10,000 to 25,000 children and adults are living with spinal muscular atrophy in the United States. It’s a rare disease that affects one out of 6,000 to 10,000 children.
Market Dynamics: Drivers
Rising adoption of novel therapeutics
The rising adoption of novel therapeutics is expected to drive the market over the forecast period. Therapies for spinal muscular atrophy are designed to interfere with the cellular basis of the disease by modifying pre-mRNA splicing and enhancing the expression of the Survival Motor Neuron (SMN) protein, which is only expressed at low levels in this disorder. Novel therapeutics are developing based on this basis, so there is an increasing demand for novel therapies and there is an increasing adoption of these therapies which leads to better patient outcomes.
For instance, in December 2023, The lab of Yongchao C. Ma, Ph.D., at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important limitations of gene therapy and other current therapies for SMA.
In addition, in December 2023, The Hospital Authority (HA) introduced a gene therapy injection for infants suffering from spinal muscular atrophy (SMA), and it is more effective than traditional treatments for the illness. It said a 10-month-old infant was the first patient in Hong Kong to receive this therapy, called onasemnogene abeparvovec.
Novel therapeutics like Zolgensma have demonstrated remarkable efficacy in clinical trials, often leading to substantial improvements in motor function and quality of life for SMA patients. This has contributed to increased confidence and adoption by healthcare professionals and patients alike. In addition, there is an ongoing clinical trial for the development of more advanced novel therapies for enhanced treatment of the condition.
For instance, on September 14, 2023, Biohaven Ltd., a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, released the completing enrollment in RESILIENT, the Phase 3 pivotal study of taldefgrobep alfa in spinal muscular atrophy (SMA).
Further, the increasing prevalence of spinal muscular atrophy, rising FDA approvals for novel therapies, increasing clinical trials to develop more advanced therapies, increasing awareness about the condition and advancements in the development of novel therapies are the factors expected to drive the market over the forecast period.
Restraints
Factors such as complications and side effects associated with the various therapies, the high cost of the treatment, unmet needs and uncertainties regarding treatment response are the factors expected to hamper the market.
Segment Analysis
The global spinal muscular atrophy treatment market is segmented based on disease type, treatment type, route of administration, end-user and region.
The disease-modifying therapy segment accounted for approximately 45.6% of the spinal muscular atrophy treatment market share
The disease-modifying therapy segment is expected to hold the largest market share over the forecast period. These disease-modifying therapy drugs stimulate the production of SMN protein. Nusinersen (Spinraza) is FDA-approved for both children and adults. The provider injects the drug into the space around the spinal canal. And risdaplam (Evrysdi), helps adults and children older than two months. People take risdaplam daily by mouth (orally).
For instance, on August 7, 2020, PTC Therapeutics, Inc. cleared the U.S. Food and Drug Administration (FDA) approval of Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. Evrysdi showed clinically meaningful improvements in motor function and obtainment of developmental milestones across two trials in patients aged two months and above and across all levels of disease severity, including types 1, 2, and 3 SMA.
In addition, in July 2021, Roche launched Evrysdi (risdiplam), an approved treatment in India for spinal muscular atrophy (SMA) patients. It is available in India within 11 months of the US approval. Since its launch, over 4,000 SMA patients across 50-plus countries have benefitted from Evrysdi. It is approved for the treatment of SMA in adults and children two months of age and older.
Further, their wide availability, acceptability and, ease of application helps to hold the largest market share. Risdiplam (Evrysdi) treatment works to stop the SMN2 genes from disrupting protein production, allowing the protein to reach the nerve cells as needed. The dosage is determined by their weight. Clinical trials showed improved muscle function after 12 months in 41% of those taking it. In addition, their wide adoption also increases the demand for the these medications.
For instance, on August 12, 2021, Chugai Pharmaceutical Co., Ltd. launched Evrysdi Dry Syrup 60 mg for the treatment of spinal muscular atrophy (SMA). This approval is based on the results from the FIREFISH study in babies with symptomatic SMA Type 1 and the SUNFISH study in children and young adults with SMA Type 2 or 3.
Geographical Analysis
North America accounted for approximately 41.4% of the market share
North America region is expected to hold the largest market share over the forecast period owing to the strong presence of major players and increasing research activities for the development of advanced therapies. North America especially the United States is very well-known for its strong presence of major players such as pharmaceutical companies. This presence of major players in the region collaborating and merging with other companies to develop more advanced medications to treat SMA.
For instance, on January 02, 2024, Voyager Therapeutics, Inc., a biotechnology company dedicated to advancing neurogenetic medicines, announced a strategic collaboration and capsid license agreement with Novartis Pharma AG to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER capsids and other intellectual property for the respective diseases.
In addition, there is an increasing research activities for the development of more advanced therapeutics for the better treatment of spinal muscular atrophy also helps to drive the market in the region. As the research activities increase, the therapeutics are developed more precisely by reducing the complications associated with the different types of therapies, resulting in better patient outcomes.
For instance, on March 14, 2022, Biogen Inc. released new data and updates from its SPINRAZA (nusinersen) and spinal muscular atrophy (SMA) research program aimed at improving clinical outcomes for people impacted by the disease, including the ASCEND, RESPOND and NURTURE studies.
Competitive Landscape
The major global players in the spinal muscular atrophy treatment market include Novartis AG, F. Hoffmann-La Roche Ltd, Genentech USA, Inc., Biogen Inc., PTC Therapeutics, Inc., Ionis Pharmaceuticals, Inc., Biohaven Ltd, Voyager Therapeutics, Inc., Catalent, Inc and Pfizer Inc. among others.
COVID-19 Impact Analysis
The COVID-19 pandemic significantly impacted the global spinal muscular atrophy treatment market. The coronavirus disease 2019 (COVID-19) pandemic has resulted in the reorganization of healthcare settings affecting the delivery of clinical care to patients with spinal muscular atrophy (SMA). There is a concern that patients with SMA may be at increased risk of manifesting severe symptoms of COVID-19. Currently approved therapies for SMA improve survival and motor function, however, their delivery requires increased exposure to the health system and a dedicated healthcare team.
Market Segmentation
By Disease Type
• Type 0 SMA (Prenatal SMA)
• Type 1 SMA (Infantile SMA, Werdnig-Hoffmann Disease)
• Type 2 SMA (Intermediate SMA, Dubowitz Disease)
• Type 3 SMA (Juvenile SMA, Kugelberg-Welander Disease)
• Type 4 SMA (Late-Onset SMA, Adults)
By Treatment Type
• Disease-Modifying Therapy
• Gene Replacement Therapy
• Others
By Route of Administration
• Intravenous
• Intrathecal
• Oral
• Others
By End-User
• Hospitals
• Specialty Clinics
• Academic and Research Institutes
• Gene Therapy Centers
• Others
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o U.K.
o France
o Spain
o Italy
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o Australia
o Rest of Asia-Pacific
• Middle East and Africa
Why Purchase the Report?
• To visualize the global spinal muscular atrophy treatment market segmentation based on disease type, treatment type, route of administration, end-user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development
• Excel data sheet with numerous data points of spinal muscular atrophy treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.
The global spinal muscular atrophy treatment market report would provide approximately 69 tables, 72 figures, and 187 Pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

상세 목차

1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Disease Type
3.2. Snippet by Treatment Type
3.3. Snippet by Route of Administration
3.4. Snippet by End-User
3.5. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Rising Adoption of Novel Therapeutics
4.1.2. Restraints
4.1.2.1. High Cost of the Treatment
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Industry Analysis
5.1. Porter's Five Force Analysis
5.2. Supply Chain Analysis
5.3. Pricing Analysis
5.4. Regulatory Analysis
5.5. Pipeline Analysis
5.6. Unmet Needs
5.7. PESTEL Analysis
5.8. Patent Analysis
5.9. SWOT Analysis
6. COVID-19 Analysis
6.1. Analysis of COVID-19
6.1.1. Scenario Before COVID
6.1.2. Scenario During COVID
6.1.3. Scenario Post COVID
6.2. Pricing Dynamics Amid COVID-19
6.3. Demand-Supply Spectrum
6.4. Government Initiatives Related to the Market During the Pandemic
6.5. Manufacturers Strategic Initiatives
6.6. Conclusion
7. By Disease Type
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
7.1.2. Market Attractiveness Index, By Disease Type
7.2. Type 0 SMA (Prenatal SMA) *
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Type 1 SMA (Infantile SMA, Werdnig-Hoffmann Disease)
7.4. Type 2 SMA (Intermediate SMA, Dubowitz Disease)
7.5. Type 3 SMA (Juvenile SMA, Kugelberg-Welander Disease)
7.6. Type 4 SMA (Late-Onset SMA, Adults)
8. By Treatment Type
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
8.1.2. Market Attractiveness Index, By Treatment Type
8.2. Disease-Modifying Therapy*
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Gene Replacement Therapy
8.4. Others
9. By Route of Administration
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
9.1.2. Market Attractiveness Index, By Route of Administration
9.2. Intravenous*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Intrathecal
9.4. Oral
9.5. Others
10. By End-User
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
10.1.2. Market Attractiveness Index, By End-User
10.2. Hospitals*
10.2.1. Introduction
10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
10.3. Specialty Clinics
10.4. Academic and Research Institutes
10.5. Gene Therapy Centers
10.6. Others
11. By Region
11.1. Introduction
11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
11.1.2. Market Attractiveness Index, By Region
11.2. North America
11.2.1. Introduction
11.2.2. Key Region-Specific Dynamics
11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.2.7.1. U.S.
11.2.7.2. Canada
11.2.7.3. Mexico
11.3. Europe
11.3.1. Introduction
11.3.2. Key Region-Specific Dynamics
11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.3.7.1. Germany
11.3.7.2. UK
11.3.7.3. France
11.3.7.4. Italy
11.3.7.5. Spain
11.3.7.6. Rest of Europe
11.4. South America
11.4.1. Introduction
11.4.2. Key Region-Specific Dynamics
11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.4.7.1. Brazil
11.4.7.2. Argentina
11.4.7.3. Rest of South America
11.5. Asia-Pacific
11.5.1. Introduction
11.5.2. Key Region-Specific Dynamics
11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.5.7.1. China
11.5.7.2. India
11.5.7.3. Japan
11.5.7.4. Australia
11.5.7.5. Rest of Asia-Pacific
11.6. Middle East and Africa
11.6.1. Introduction
11.6.2. Key Region-Specific Dynamics
11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
12. Competitive Landscape
12.1. Competitive Scenario
12.2. Market Positioning/Share Analysis
12.3. Mergers and Acquisitions Analysis
13. Company Profiles
13.1. Novartis AG*
13.1.1. Company Overview
13.1.2. Product Portfolio and Description
13.1.3. Financial Overview
13.1.4. Key Developments
13.2. F. Hoffmann-La Roche Ltd
13.3. Genentech USA, Inc.
13.4. Biogen Inc.
13.5. PTC Therapeutics, Inc.
13.6. Ionis Pharmaceuticals, Inc.
13.7. Biohaven Ltd
13.8. Voyager Therapeutics, Inc.
13.9. Catalent, Inc
13.10. Pfizer Inc.
LIST NOT EXHAUSTIVE
14. Appendix
14.1. About Us and Services
14.2. Contact Us

언급된 주요 기업들

Novartis AG, 4.	Key Developments, F. Hoffmann-La Roche Ltd, Genentech USA, Inc., Biogen Inc., PTC Therapeutics, Inc., Ionis Pharmaceuticals, Inc., Biohaven Ltd, Voyager Therapeutics, Inc., Catalent, Inc, Pfizer Inc.

표 목록 (Tables)

List of Tables

Table 1 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2023, 2027 & 2031 (US$ Million)

Table 2 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2023, 2027 & 2031 (US$ Million)

Table 3 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2023, 2027 & 2031 (US$ Million)

Table 4 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2023, 2027 & 2031 (US$ Million)

Table 5 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Region, 2023, 2027 & 2031 (US$ Million)

Table 6 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2023, 2027 & 2031 (US$ Million)

Table 7 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2022-2031 (US$ Million)

Table 8 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2023, 2027 & 2031 (US$ Million)

Table 9 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2022-2031 (US$ Million)

Table 10 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2023, 2027 & 2031 (US$ Million)

Table 11 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2022-2031 (US$ Million)

Table 12 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2023, 2027 & 2031 (US$ Million)

Table 13 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2022-2031 (US$ Million)

Table 14 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Region, 2023, 2027 & 2031 (US$ Million)

Table 15 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, By Region, 2022-2031 (US$ Million)

Table 16 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2022-2031 (US$ Million)

Table 17 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2022-2031 (US$ Million)

Table 18 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2022-2031 (US$ Million)

Table 19 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2022-2031 (US$ Million)

Table 20 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Country, 2022-2031 (US$ Million)

Table 21 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2022-2031 (US$ Million)

Table 22 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2022-2031 (US$ Million)

Table 23 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2022-2031 (US$ Million)

Table 24 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2022-2031 (US$ Million)

Table 25 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, By Country, 2022-2031 (US$ Million)

Table 26 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2022-2031 (US$ Million)

Table 27 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2022-2031 (US$ Million)

Table 28 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2022-2031 (US$ Million)

Table 29 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2022-2031 (US$ Million)

Table 30 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, By Country, 2022-2031 (US$ Million)

Table 31 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2022-2031 (US$ Million)

Table 32 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2022-2031 (US$ Million)

Table 33 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2022-2031 (US$ Million)

Table 34 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2022-2031 (US$ Million)

Table 35 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, By Country, 2022-2031 (US$ Million)

Table 36 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Value, By Disease Type, 2022-2031 (US$ Million)

Table 37 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Value, By Treatment Type, 2022-2031 (US$ Million)

Table 38 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Value, By Route of Administration, 2022-2031 (US$ Million)

Table 39 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Value, By End-User, 2022-2031 (US$ Million)

Table 40 Novartis AG: Overview

Table 41 Novartis AG: Product Portfolio

Table 42 Novartis AG: Key Developments

Table 43 F. Hoffmann-La Roche Ltd: Overview

Table 44 F. Hoffmann-La Roche Ltd: Product Portfolio

Table 45 F. Hoffmann-La Roche Ltd: Key Developments

Table 46 Genentech USA, Inc.: Overview

Table 47 Genentech USA, Inc.: Product Portfolio

Table 48 Genentech USA, Inc.: Key Developments

Table 49 Biogen Inc.: Overview

Table 50 Biogen Inc.: Product Portfolio

Table 51 Biogen Inc.: Key Developments

Table 52 PTC Therapeutics, Inc.: Overview

Table 53 PTC Therapeutics, Inc.: Product Portfolio

Table 54 PTC Therapeutics, Inc.: Key Developments

Table 55 Ionis Pharmaceuticals, Inc.: Overview

Table 56 Ionis Pharmaceuticals, Inc.: Product Portfolio

Table 57 Ionis Pharmaceuticals, Inc.: Key Developments

Table 58 Biohaven Ltd: Overview

Table 59 Biohaven Ltd: Product Portfolio

Table 60 Biohaven Ltd: Key Developments

Table 61 Voyager Therapeutics, Inc.: Overview

Table 62 Voyager Therapeutics, Inc.: Product Portfolio

Table 63 Voyager Therapeutics, Inc.: Key Developments

Table 64 Catalent, Inc: Overview

Table 65 Catalent, Inc: Product Portfolio

Table 66 Catalent, Inc: Key Developments

Table 67 Pfizer Inc.: Overview

Table 68 Pfizer Inc.: Product Portfolio

Table 69 Pfizer Inc.: Key Developments

그림 목록 (Figures)

List of Figures

Figure 1 Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 2 Global Spinal Muscular Atrophy (SMA) Treatment Market Share, By Disease Type, 2022 & 2031 (%)

Figure 3 Global Spinal Muscular Atrophy (SMA) Treatment Market Share, By Treatment Type, 2022 & 2031 (%)

Figure 4 Global Spinal Muscular Atrophy (SMA) Treatment Market Share, By Route of Administration, 2022 & 2031 (%)

Figure 5 Global Spinal Muscular Atrophy (SMA) Treatment Market Share, By End-User, 2022 & 2031 (%)

Figure 6 Global Spinal Muscular Atrophy (SMA) Treatment Market Share, By Region, 2022 & 2031 (%)

Figure 7 Global Spinal Muscular Atrophy (SMA) Treatment Market Y-o-Y Growth, By Disease Type, 2022-2031 (%)

Figure 8 Type 0 SMA (Prenatal SMA) Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 9 Type 1 SMA (Infantile SMA, Werdnig-Hoffmann Disease) Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 10 Type 2 SMA (Intermediate SMA, Dubowitz Disease) Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 11 Type 3 SMA (Juvenile SMA, Kugelberg-Welander Disease) Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 12 Type 4 SMA (Late-Onset SMA, Adults) Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 13 Global Spinal Muscular Atrophy (SMA) Treatment Market Y-o-Y Growth, By Treatment Type, 2022-2031 (%)

Figure 14 Disease-Modifying Therapy Treatment Type in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 15 Gene Replacement Therapy Treatment Type in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 16 Others Treatment Type in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 17 Global Spinal Muscular Atrophy (SMA) Treatment Market Y-o-Y Growth, By Route of Administration, 2022-2031 (%)

Figure 18 Intravenous Route of Administration in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 19 Intrathecal Route of Administration in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 20 Oral Route of Administration in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 21 Others Route of Administration in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 22 Global Spinal Muscular Atrophy (SMA) Treatment Market Y-o-Y Growth, By End-User, 2022-2031 (%)

Figure 23 Hospitals End-User in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 24 Specialty Clinics End-User in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 25 Academic and Research Institutes End-User in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 26 Gene Therapy Centers End-User in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 27 Others End-User in Global Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 28 Global Spinal Muscular Atrophy (SMA) Treatment Market Y-o-Y Growth, By Region, 2022-2031 (%)

Figure 29 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 30 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 31 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 32 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 33 Middle East and Africa Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 34 North America Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 35 North America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Disease Type, 2022 & 2031 (%)

Figure 36 North America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Treatment Type, 2022 & 2031 (%)

Figure 37 North America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Route of Administration, 2022 & 2031 (%)

Figure 38 North America Spinal Muscular Atrophy (SMA) Treatment Market Share, By End-User, 2022 & 2031 (%)

Figure 39 North America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Country, 2022 & 2031 (%)

Figure 40 South America Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 41 South America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Disease Type, 2022 & 2031 (%)

Figure 42 South America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Treatment Type, 2022 & 2031 (%)

Figure 43 South America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Route of Administration, 2022 & 2031 (%)

Figure 44 South America Spinal Muscular Atrophy (SMA) Treatment Market Share, By End-User, 2022 & 2031 (%)

Figure 45 South America Spinal Muscular Atrophy (SMA) Treatment Market Share, By Country, 2022 & 2031 (%)

Figure 46 Europe Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 47 Europe Spinal Muscular Atrophy (SMA) Treatment Market Share, By Disease Type, 2022 & 2031 (%)

Figure 48 Europe Spinal Muscular Atrophy (SMA) Treatment Market Share, By Treatment Type, 2022 & 2031 (%)

Figure 49 Europe Spinal Muscular Atrophy (SMA) Treatment Market Share, By Route of Administration, 2022 & 2031 (%)

Figure 50 Europe Spinal Muscular Atrophy (SMA) Treatment Market Share, By End-User, 2022 & 2031 (%)

Figure 51 Europe Spinal Muscular Atrophy (SMA) Treatment Market Share, By Country, 2022 & 2031 (%)

Figure 52 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 53 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Share, By Disease Type, 2022 & 2031 (%)

Figure 54 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Share, By Treatment Type, 2022 & 2031 (%)

Figure 55 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Share, By Route of Administration, 2022 & 2031 (%)

Figure 56 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Share, By End-User, 2022 & 2031 (%)

Figure 57 Asia-Pacific Spinal Muscular Atrophy (SMA) Treatment Market Share, By Country, 2022 & 2031 (%)

Figure 58 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Value, 2022-2031 (US$ Million)

Figure 59 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Share, By Disease Type, 2022 & 2031 (%)

Figure 60 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Share, By Treatment Type, 2022 & 2031 (%)

Figure 61 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Share, By Route of Administration, 2022 & 2031 (%)

Figure 62 Middle East & Africa Spinal Muscular Atrophy (SMA) Treatment Market Share, By End-User, 2022 & 2031 (%)

Figure 63 Novartis AG: Financials

Figure 64 F. Hoffmann-La Roche Ltd: Financials

Figure 65 Genentech USA, Inc.: Financials

Figure 66 Biogen Inc.: Financials

Figure 67 PTC Therapeutics, Inc.: Financials

Figure 68 Ionis Pharmaceuticals, Inc.: Financials

Figure 69 Biohaven Ltd: Financials

Figure 70 Voyager Therapeutics, Inc.: Financials

Figure 71 Catalent, Inc: Financials

Figure 72 Pfizer Inc.: Financials

세계 척수근육위축증(SMA) 치료 시장 – 2023-2030

Global Spinal Muscular Atrophy (SMA) Treatment Market - 2023-2030